Clinical trials are crucial whenever a new drug, therapy, medical procedure or intervention is being developed. This is because every prospective treatment must be rigorously tested before it can be deemed safe and usable. 

 

But with so much at stake, these trails have to be meticulously thought-out and designed to ensure the most accurate and insightful results. 

 

However, designing and running a clinical trial is no small job. It is a complex process that requires careful planning to ensure the study is scientifically valid, ethical and capable of answering the original research question. 

 

Here’s a step-by-step guide on how to get it right. 

 

Carefully define the research question or objective 

 

The first important step is to determine the primary objective of the study, for example, whether you are testing a new type of drug or medical procedure. This can lead you to a clear, testable hypothesis and parameters for the study. 

 

Your hypothesis cannot be a vague concept, such as, ‘does this work’? It must be as clear and well-defined as possible in order to identify the appropriate type of trial to use and the necessary components to test the question and get the most accurate answers.

 

Conduct a literature review 

 

Before you dive in, it’s important to review any existing studies or trials that are closely related to your own. This will enable you to identify any gaps in the current knowledge, which can help you to refine your research question even further. 

 

This also presents you with the opportunity to ensure that your study is novel and feasible based on existing evidence or past trials. 

 

Determine the trial population

 

The next step is to define the characteristics that all participants must have in order to be included in the study, such as age range, gender, disease state, etc. For example, a trial on the effectiveness of heart medication for men over 60 will not achieve the right results if tested on a population of young women.

 

At this stage, it’s also important to choose a control group. How they are controlled will be determined by the type of trial you are designing and what you are testing, but more on this later. 

 

Understand and plan each phase carefully and correctly

 

Most clinical trials will take place through four key phases, so it’s vital that you plan each of these phases carefully. Be aware that this is not always the case, but for trials testing new drugs or treatments, the phases are likely to work as follows: 

 

Phase 1: This is when a smaller group of healthy participants are used to assess the safety, side effects and effectiveness of the new drug or intervention. Not every trial will continue after this stage depending on the early results. 

 

Phase 2: The next stage is randomized trials of a group of participants with a related illness or condition, this determines the efficacy of the product or treatment being tested. 

 

Phase 3: Now you can continue with a much larger group of participants (still those with the related illness or condition) using a randomized and blind testing study. This is where the data pool really starts to expand. 

 

Phase 4: This phase is an ongoing study in order to determine any long-term effects and to gauge the safety of the product or treatment for use long into the future. 

 

Whether or not the design of your trial will follow this exact pattern will very much depend on what is being tested, but it’s important to understand each of these phases and plan for them accordingly. 

 

Decide on your sample size

 

As the different phases require different types and numbers of participants, you need to decide on your sample size and make sure you’re going to have enough people taking part. 

 

In order to do this, you need to conduct a detailed analysis to determine the required sample size for your study. The sample size must be large enough to generate impactful results and reduce the risk of bias and errors.

 

Define the intervention and control groups 

 

There are several different types of clinical trials including a randomized controlled trial (RCT), cohort study or cross-over trials.

 

Which style you choose will determine the intervention and the treatment or intervention being tested on them. It will also determine the control, whether that’s a placebo, standard treatment or no treatment at all. 

 

It’s important to know at this stage that blinding must occur in order to achieve accurate results. This ensures that the participants don’t know what their assigned group or intervention is, this can prevent bias, behavioral changes or placebo effects that could damage the end results.

 

Make sure you have the budget and backing 

 

In addition to having enough participants, you also need to make sure you have the budget and backing to keep the trial going. So, at the design phase, it’s crucial to set a budget and assess how and where this will be spent – even if this is just an estimate. 

 

It’s also important that you have a robust and diverse group of stakeholders. This may include researchers, regulators, scientists, doctors, sponsors and more. 

 

Make sure that you’ve got all the backing you need in order to effectively conduct, complete and analyze the study before you begin.

 

Set primary endpoints 

 

Your trial needs to set endpoints that define the main outcome(s) and what is measured in order to determine the effect of the intervention, such as survival rate or symptom improvement. You need to set your primary endpoints during the initial design process. 

 

However, you might also find that as the study unfolds, secondary endpoints become apparent that identify additional outcomes of interest.

 

Develop a protocol and ensure regulatory compliance

 

One of the best ways to ensure your trial is designed and executed correctly is to write a detailed plan. You should put together a comprehensive protocol that outlines every aspect of the trial that we’ve discussed so far, including objectives, methodology, budget, statistical analysis and ethical considerations. 

 

This will also help with regulatory compliance. You must ensure that the protocol complies with all regulatory standards and guidelines, as well as ethical considerations and data management laws. 

 

Within your protocol, outline your plan for collecting and storing data, including measures for data protection and participant confidentiality. This is vital for the legitimacy of the study and avoiding any legal battles later on.

 

Decide on the right tools

 

Lastly, as technology continues to evolve, there are lots of useful tools that can help with the design and execution of your clinical trial. For example, virtual advisory boards that allow all stakeholders and participants to communicate and interact seamlessly. 

 

For the purpose of designing your trial, there are also simulation tools that enable you to test your trial strategy ahead of time to spot any issues or weaknesses and tackle these before the trial begins. 

 

It is recommended that you test your design before you begin with the trial to avoid problems, budgetary issues and ultimately, a failed trial. 

 

By researching and investing in the right tools and outlining where these will be used in the trial design you can increase regulatory approvals, reduce costs and get faster results.